FDA outlines new review pathway for drugs treating ultra-rare diseases

BioPharma Dive September 4, 2025
Ned Pagliarulo

The agency’s two main review offices will work together to flexibly evaluate medicines for serious conditions that affect fewer than 1,000 people in the U.S.

Dive Brief:

• The Food and Drug Administration plans to work more closely and flexibly with developers of drugs for certain ultra-rare conditions, outlining Wednesday a new process to coordinate its assessment of such medicines across the agency’s two principal review offices.
• The Center for Drug Evaluation and Research will work together with the Center for Biologics Evaluation and Research to jointly oversee the new process, which the FDA is calling Rare Disease Evidence Principles, or RDEP.
• Under RDEP, the FDA plans to accept evidence generated by single-arm clinical trials, along with supportive evidence...

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